Milademetan is a Small Molecule owned by Rain Therapeutics, and is involved in 9 clinical trials, of which 5 were completed, 3 are ongoing, and 1 is planned.

Milademetan inhibits murine double minute 2 (MDM2). MDM2, a zinc finger protein, is a negative regulator of the p53 pathway; often over-expressed in cancer cells. It has been implicated in cancer cell proliferation and survival. The drug candidate binds to MDM2, thereby preventing the binding of the MDM2 protein to the transcriptional activation domain of the tumor suppressor protein p53. By preventing this MDM2-p53 interaction, the proteosome-mediated enzymatic degradation of p53 is inhibited and the transcriptional activity of p53 is restored, which may result in the restoration of p53 signaling and thus the p53-mediated induction of tumor cell apoptosis.

The revenue for Milademetan is expected to reach a total of $820m through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Milademetan NPV Report.

Milademetan was originated by Rigel Pharmaceuticals and is currently owned by Rain Therapeutics.

Milademetan Overview

Milademetan is under development for the treatment of advanced solid tumors, liposarcoma, dedifferentiated liposarcoma, refractory or relapsed acute myeloid leukemia (AML),  pancreatic cancer, lung cancer, breast cancer, biliary tumor and malignant pleural mesothelioma (MPM). The drug candidate is administered orally as a capsule. The drug candidate acts by targeting murine double minute 2 (MDM2) protein.

It was also under development for acute lymphocytic leukemia (ALL), Merkel cell carcinoma, multiple myeloma, soft tissue sarcoma, lymphomas, chronic myelogenous leukemia (CML) and myelodysplastic syndrome.

Rain Therapeutics Overview

Rain Therapeutics is a biotechnology company that discovers, develops and translates cancer therapies. It is investigating its lead product candidate milademetan, an oral small molecule, inhibitor of mouse double minute 2 (MDM2) to treat well-differentiated (WD) and dedifferentiated (DD) liposarcoma, Merkel cell carcinoma, amplified and advanced solid tumors. The company is also evaluating a RAD52 inhibiting program targeting homologous recombination deficiencies (HRD+) and loss of function of several pathway constituents including BRCA1/2, PALB2 in breast, prostate, pancreatic, ovarian and other tumors. Rain Therapeutics is headquartered in Newark, California, the US.

The operating loss of the company was US$51.5 million in FY2021, compared to an operating loss of US$19 million in FY2020. The net loss of the company was US$51.4 million in FY2021, compared to a net loss of US$21.1 million in FY2020.

Quick View – Milademetan

Report Segments
  • Innovator
Drug Name
  • Milademetan
Administration Pathway
  • Oral
Therapeutic Areas
  • Oncology
Key Companies
Highest Development Stage
  • Phase III

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.